Vertex presents dossier to the EMA to add around 200 rare CFTR mutations to the Kaftrio License
If approved by the EMA, around 2,800 people with CF across the EU, including Ireland, aged 2 years and above could benefit
Vertex have recently issued a press release (24 November 2023) which will be of much interest to those with rarer CFTR mutations in Ireland and across the EU.
Vertex have submitted a dossier (application) to the European Medicines Agency (EMA) advocating for the inclusion of 200 rarer (non-F508del CFTR mutations) be added to the license for Kaftrio aged two years and above. These are mutations that have proved responsive in clinical trials and/or ‘in vitro’ (test tube, culture dish, or elsewhere outside a living organism - including organoid testing - if backed up with other evidence).
The Vertex dossier to the EMA also includes the types of mutations that are already covered in the FDA license in the US.
Philip Watt CEO of Cystic Fibrosis Ireland stated:
"This is a really welcome first step for those people with CF that currently do not get access to Kaftrio because of their rare CFTR mutation is currently not approved by the EMA. It will take some months now for the Vertex dossier to be considered by the EMA and all being well, sanctioned by the European Commission and the Irish Government. In welcoming this crucial first step – it’s also important to emphasise that some people will never benefit (or may not tolerate) a CFTR modulator - so there is still need for new and innovative other therapies. The potential of mRNA and gene editing are examples of innovative therapies being researched at present- with still no definitive research outcomes from either and more research needed."
"No one with CF left behind"